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182 articles for “Gene therapy”
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A review on Design of Gene Therapy and Its Potential for Craniofacial Regeneration
Abstract: One of the most talked-about topics of the 21st century is gene therapy, which holds the promise of treating many diseases. Current gene therapy research explores a wide range of potential treatments, such as enhancing the body’s immune response to tumors, promoting the formation of new blood vessels in the heart to mitigate heart attacks, and preventing HIV replication in AIDS patients. Gene therapy involves the introduction, alteration, or replacement …
Published in Emerging Trends in Personalized Medicines · Vol. 2, Issue 2, 2025 · pp. 6–12 Read article
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Innovations in Gene Therapy: Revolutionizing Treatment for Genetic Disorders and Diseases
Abstract: Gene therapy is a revolutionary intervention in the treatment of genetic diseases since it is an intervention that aims at the very source of diseases, through editing and modification of genes. More recent innovations in gene editing systems, such as CRISPR-Cas9, CRISPR-Cas12, and prime editing, have greatly increased the accuracy and effectiveness of gene therapies, enabling more specific therapies. The opportunities of personalized medicine are also extended by the creation …
Published in Research and Reviews : A Journal of Biotechnology · Vol. 16, Issue 1, 2026 Read article
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Gene Therapy in Modern Medicine: Promises and Challenge in Treating Genetic Diseases
Abstract: Gene therapy is a medical approach that focuses on altering or adjusting an individual’s genes to treat or prevent illnesses. The aim is to repair faulty genes or insert new ones into the body to combat diseases. Gene therapy can involve directly introducing modified or new genes into a patient’s cells or altering the genes already present in the patient’s body. This approach shows potential for treating a range of …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 2, Issue 2, 2024 · pp. 27–32 Read article
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An Overview of Gene Therapy
Abstract: Gene therapy is a revolutionary technique in medical science that seeks to cure or stop illnesses by means of introducing, altering, or quieting genes inside a patient's cells. Advancements in molecular biology, genetics, and biotechnology have propelled this field forward markedly changed over the last few decades. The underlying idea of gene therapy is to introduce genetic material into target cells via viral or non-viral vectors to rectify faulty genes, …
Published in Research and Reviews: A Journal of Pharmaceutical Science · Vol. 16, Issue 3, 2025 · pp. 89–105 Read article
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Gene Therapy 2.0: Harnessing Cutting-Edge Technologies for Precision Medicine
Abstract: Somatic gene therapy has become the standard form of gene therapy; this creates the Gene Therapy 2.0 era. This new and distinct time consists in the new-generation genome-editing tools and markers, more efficient systems for gene delivery, and personalized medicine approaches that raise the efficacy and safety of the treatment. Base and prime editing are two types of CRISPR-based gene editing tools have provided high precision and reduced off-target effects …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 2, 2025 Read article
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Advancing Gene Therapy: Next-Generation Viral Vector Engineering for Precision, Safety, and Scalability
Abstract: Gene therapy has emerged as a paradigm-shifting modality for the treatment of genetic disorders, malignancies, and rare diseases through the delivery of therapeutic nucleic acids aimed at correcting or modulating dysfunctional gene expression. Among the various delivery systems, viral vectors including adeno-associated viruses (AAVs), lentiviruses, adenoviruses, retroviruses, and herpes simplex viruses have proven indispensable owing to their high transduction efficiencies and adaptability. This review offers a comprehensive assessment of viral …
Published in International Journal of Virus Studies · Vol. 2, Issue 2, 2025 · pp. 37–55 Read article
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Genetic Counseling: A Pathway to Understanding and Managing Inherited Health Risks
Abstract: Genetic counseling is an essential aspect of personalized medicine that supports individuals and families in understanding the implications of inherited health conditions. With the rising prevalence of genetic disorders and advancements in genomic technologies, counseling provides critical insights into risk factors, diagnostic options, and informed decision-making. This paper explores the classification of genetic disorders, major genetic and environmental causes, and the expanding role of genetic counseling within India’s culturally diverse …
Published in International Journal of Evidence Based Nursing And Practices · Vol. 4, Issue 1, 2026 · pp. 10–14 Read article
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Wiskott-Aldrich Syndrome: A Comprehensive Guide
Abstract: Wiskott-Aldrich Syndrome (WAS) is an uncommon genetic disorder inherited through the X chromosome, marked by a combination of immune system deficiencies, eczema, and low platelet counts. This syndrome primarily affects males, leading to significant morbidity and mortality due to recurrent infections, bleeding complications, and autoimmune diseases. Mutations in the WAS gene disrupt the production of the Wiskott-Aldrich Syndrome protein (WASp), crucial for the functioning of immune cells and platelet formation. …
Published in Research and Reviews : A Journal of Immunology · Vol. 14, Issue 2, 2024 · pp. 39–46 Read article
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Emerging Therapies in Respiratory Tract Infections: Current Advances and Future Perspectives
Abstract: The impact of respiratory tract infections (RTI) as a global public health problem continues to be high morbidity and mortality rates. The development of novel therapeutic approaches stemmed from the fact that conventional treatment strategies, antibiotics, antivirals, antifungals etc. have become increasingly ineffective because of rise of antimicrobial resistance (AMR). Next generation therapies, such as novel antimicrobial agents, host directed therapies and novel drug delivery systems have come on the …
Published in Recent Trends in Infectious Diseases · Vol. 2, Issue 2, 2025 · pp. 17–25 Read article
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Carboxymethyl Cellulose-Based Spray-Dried Microspheres: Recent Advances and Applications in Targeted Drug Delivery Systems
Abstract: Microspheres are spherical particles ranging from 1 to 1000 μm, made from natural or synthetic polymers and inorganic materials. Their structure allows precise drug delivery, improving targeted release and minimizing off-target effects. Natural polymers such as starch, chitosan, and alginate are favored for their biodegradability, adhesion, and compatibility, enhancing mucosal interaction and residence time. Microspheres are widely used in site-specific drug delivery, gene therapy, and vaccine administration, improving immunogenicity, extending …
Published in Journal of Polymer & Composites · Vol. 13, Issue 5, 2025 · pp. 251–268 Read article
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An AAV Vector for Inducible Gene Expression Preferentially in Muscles
Abstract: Adeno associated viral (AAV) vectors has been used widely in gene therapy and efforts have been made to improve their utility by adding genetic elements that would enable targeting transgene expression to particular cells or tissues of interest and permitting on/off regulation of expression. In this study, we designed a recombinant AAV9 variant PHP.eB vector with muscle creatine kinase (Mck)-derived enhancers, a synthetic muscle-expression promoter, in combination with a third-generation …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 1, Issue 2, 2023 · pp. 46–61 Read article
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CRISPR-Cas9: Revolutionizing the Genetic Frontier
Abstract: The rapid development of CRISPR/CRISPR-associated enzyme (Cas) technology has enabled truly customised treatment of human genetic disorders, paving the way for recent developments in the field of gene therapy. Because CRISPR/Cas can accurately target and edit individual genes within a genome, it has established itself as a formidable tool for genetic manipulation. CRISPR/Cas9 technology allows for precise editing of specific DNA sequences in an organism's genome. This method uses three …
Published in International Journal of Genetic Modifications and Recombinations · Vol. 3, Issue 2, 2025 Read article
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Biological Membranes: Structure, Function, and Their Roles in Disease Pathophysiology
Abstract: Biological membranes are important structures that control cellular work, signal transmission, transport, and communication. Dysfunction of membranes has been considered in the development of many diseases, such as cancer, neurodegenerative diseases, and infections, with the distortion of lipid composition, protein activity, and cellular connections involved in the pathogenesis of the disease. The damage on membrane can be caused by oxidative stress, genetic mutation, and environmental influence to disrupt cellular homeostasis. …
Published in International Journal of Membranes · Vol. 3, Issue 1, 2026 · pp. 38–46 Read article
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Progeria Syndrome Unveiled: A Scientific Odyssey into Premature Aging Mechanisms and Therapeutic Frontiers
Abstract: Progeria, or Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare and fatal genetic disorder in childhood, exhibiting features akin to premature aging. Despite normal appearances in infancy, affected children face accelerated aging with distinct facial characteristics, including micrognathia, dental malformations, lower body weight, early hair loss, decreased joint mobility, lipodystrophy, etc. The cause of HGPS is a point mutation that occurs at the exon 11 of the LMNA gene which normally …
Published in Research and Reviews: A Journal of Health Professions · Vol. 14, Issue 2, 2024 · pp. 52–68 Read article
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RNA- Based Drug Delivery, Current Advances, Pharmaceutical Applications, and Future Perspectives
Abstract: RNA-based therapeutics have emerged as a transformative approach in modern medicine due to their ability to regulate gene expression with high specificity and precision. Advances in molecular biology, RNA chemistry, and nanotechnology have accelerated the development of diverse RNA modalities, including messenger RNA (mRNA), small interfering RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASOs), and RNA aptamers. These therapeutics offer promising strategies for the treatment of cancer, genetic disorders, infectious diseases, …
Published in Research & Reviews: A Journal of Drug Design & Discovery · Vol. 13, Issue 2, 2026 Read article
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Exhaustive Search Meets DNA Sequencing: A Comprehensive Review of TSP-Based Approaches
Abstract: Exhaustive search is a highly computational complex algorithm that checks every possibility to obtain the best solution. We illustrate an exhaustive search by applying it to three important problems: the traveling salesman problem, the knapsack problem, and the assignment problem. In this paper, we took a traveling salesman problem to explain DNA Sequencing. Since traveling salesman problem is an algorithmic problem that finds the shortest route between a set of …
Published in International Journal of Bioinformatics and Computational Biology · Vol. 2, Issue 2, 2024 · pp. 11–21 Read article
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Overcoming Obstacles to an HIV-1 Cure: Advances in Genome Editing, Immune System Reprogramming, and Viral Reservoir Elimination
Abstract: Human immunodeficiency virus type-1 (HIV-1) continues to represent a major global health challenge despite the remarkable success of antiretroviral therapy (ART). Although ART effectively suppresses viral replication and prolongs survival, it cannot eradicate the virus due to the persistence of long-lived latent reservoirs. In recent years, innovative therapeutic strategies have been explored with the aim of achieving either a functional remission or complete viral elimination. Advances in genome-editing technologies, particularly …
Published in International Journal of Virus Studies · Vol. 3, Issue 2, 2026 Read article
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Magnetic and Radioactive Nanoparticles for Improved Theranostics and AI Assisted Radiation Therapy
Abstract: Nanoparticles based therapeutic and theranostics technique is becoming an active area of research in nanomedicine. The sensitivity, biocompatibility, and stability of magnetic and radioactive nanoparticles determine their functionality. This research highlights the impacts of magnetic and radioactive nanoparticles on therapeutic techniques namely, cell therapy, gene therapy, and tissue regeneration. Then, it is intended to brief a principal role of these therapeutic techniques to envisage theranostics medicine and radiation therapy using …
Published in International Journal of Advance in Molecular Engineering · Vol. 3, Issue 2, 2025 · pp. 10–21 Read article
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Development of a Novel Therapeutic Approach Using RNA Interference for Targeting Viral Genes
Abstract: RNA interference (RNAi) is a ground breaking therapeutic strategy that holds immense promise for targeting viral genes in the treatment of viral infections. This approach leverages the natural gene-silencing mechanisms of RNAi to inhibit viral replication and expression at the molecular level, providing a precise and adaptable method to counteract viral pathogens, including influenza, HIV, and hepatitis viruses. Recent advancements in RNAi technology have enabled the development of highly specific …
Published in International Journal of Virus Studies · Vol. 1, Issue 2, 2024 · pp. 12–22 Read article
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Revolutionizing Blood Cancer Treatment: Emerging Therapies and Future Perspectives
Abstract: Blood cancer inclusive of leukemia, lymphoma and multiple myeloma poses a significant threat in the world today because of its multifactorial process and its non-amenable to standard therapies. Some of such approaches are immunotherapy, gene-editing technology and targeted small molecule inhibitors that have altered the nature of treatments. CAR T cell therapy is a molecular targeted therapy for cancers and several clinical studies have reported a huge success. Also, the …
Published in International Journal of Biomedical Innovations and Engineering · Vol. 3, Issue 2, 2025 Read article